MeiraGTx Announces Investigational Gene Therapy Continues to Demonstrate Statistically Significant Improvement in Vision in Patients with X-Linked Retinitis Pigmentosa One Year After Treatment

Data presented at AAO 2020 Virtual Annual Meeting show sustained improvements in retinal sensitivity at 12 months

LONDON and NEW YORK, Nov. 13, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced 12-month data from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy in development for the treatment of X-linked retinitis pigmentosa (XLRP). Statistically significant vision improvement in the dose escalation phase of the trial was sustained one year after treatment. These data were presented today as a late-breaker oral presentation at the American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In the ongoing Phase 1/2 MGT009 clinical trial, each patient was treated with subretinal delivery of AAV-RPGR in one eye and the patient’s other eye served as an untreated control. The primary endpoint of the trial is safety, with secondary endpoints assessing changes in visual function at pre-specified timepoints post-treatment. Baseline values were determined in triplicate.

In the dose escalation phase of the trial, data at the 12-month time point demonstrated statistically significant improvement in retinal sensitivity in treated eyes in both the low (n=3) and intermediate (n=4) dose cohorts, with six of seven patients demonstrating improved or stable vision in the treated eye one year after treatment.

“Significant vision improvement in XLRP patients was sustained one year after treatment with this novel gene therapy,” said Michel Michaelides¹, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. “XLRP is a degenerative disease that progresses to blindness in working-age adults. These findings suggest AAV-RPGR has the potential to not only stabilize vision, but to actually improve vision in patients who have no current therapeutic options.”